Guide To Human Gene Therapy, A

Roland W Herzog, Sergei Zolotukhin

• Publisher: World Scientific
• Year: 2010
• Print ISBN: 9789814280907, 9789814280907
• eBook ISBN: 9789814280914
• Topic: Biological Sciences
• Subtopic: Science General
• Index: Biological Sciences

Ever since the birth of molecular biology, the tantalizing possibility of treating disease at its genetic roots has become increasingly feasible. Gene therapy — though still in its infancy — remains one of the hottest areas of research in medicine. Its approach utilizes a gene transfer vehicle ('vector') to deliver therapeutic DNA or RNA to cells of the body in order to rectify the defect that is causing the disease. Successful therapies have been reported in humans in recent years such as cures in boys with severe immune deficiencies. Moreover, gene therapy strategies are being adapted in numerous biomedical laboratories to obtain novel treatments for a variety of diseases and to study basic biological aspects of disease. Correction of disease in animal studies, is steadily gaining ground, highlighting the immense potential of gene therapy in the medical profession.This book will cover topics that are at the forefront of biomedical research such as RNA interference, viral and non-viral gene transfer systems, treatment of hematological diseases and disorders of the central nervous system. Leading experts on the respective vector or disease will contribute the individual chapters and explain cutting-edge technologies. It also gives a broad overview of the most important gene transfer vectors and most extensively studied target diseases. This comprehensive guide is therefore a must-read for anyone in the biotechnology, biomedical or medical industries seeking to further their knowledge in the area of human gene therapy.

Table of contents

• Contents
• Preface
• Contributors
• 1. Non-Viral Gene Therapy Sean M. Sullivan
• 2. Adenoviral Vectors Stuart A. Nicklin and Andrew H. Baker
• 3. Retroviral Vectors and Integration Analysis Cynthia C. Bartholomae, Romy Kirsten, Hanno Glimm, Manfred Schmidt and Christof von Kalle
• 4. Lentiviral Vectors Janka Mátrai, Marinee K. L. Chuah and Thierry VandenDriessche
• 5. Herpes Simplex Virus Vectors William F. Goins, David M. Krisky, James B. Wechuck, Darren Wolfe, Justus B. Cohen and Joseph C. Glorioso
• 6. Adeno-Associated Viral (AAV) Vectors Nicholas Muzyczka
• 7. Regulatory RNA in Gene Therapy Alfred. S. Lewin
• 8. DNA Integrating Vectors (Transposon, Integrase) Lauren E. Woodard and Michele P. Calos
• 9. Homologous Recombination and Targeted Gene Modification for Gene Therapy Matthew Porteus
• 10. Gene Switches for Pre-Clinical Studies in Gene Therapy Caroline Le Guiner, Knut Stieger, Alice Toromanoff, Fabienne Rolling, Philippe Moullier and Oumeya Adjali
• 11. Gene Therapy for Central Nervous System Disorders Deborah Young and Patricia A. Lawlor
• 12. Gene Therapy of Hemoglobinopathies Angela E. Rivers and Arun Srivastava
• 13. Gene Therapy for Primary Immunodeficiencies Aisha Sauer, Barbara Cassani and Alessandro Aiuti
• 14. Gene Therapy for Hemophilia David Markusic, Babak Moghimi and Roland Herzog
• 15. Gene Therapy for Obesity and Diabetes Sergei Zolotukhin and Clive H. Wasserfall
• 16. Gene Therapy for Duchenne Muscular Dystrophy Takashi Okada and Shin’ichi Takeda
• 17. Cancer Gene Therapy Kirsten A.K. Weigel-Van Aken
• 18. Gene Therapy for Autoimmune Disorders Daniel F. Gaddy, Melanie A. Ruffner and Paul D. Robbins
• 19. Gene Therapy for Inherited Metabolic Storage Diseases Cathryn Mah
• 20. Retinal Diseases Shannon E. Boye, Sanford L. Boye and William W. Hauswirth
• 21. A Brief Guide to Gene Therapy Treatments for Pulmonary Diseases Ashley T. Martino, Christian Mueller and Terence R. Flotte
• 22. Cardiovascular Disease Darin J. Falk, Cathryn S. Mah and Barry J. Byrne
• Index